Could lasers make stem cell therapy available to everyone? : NPR



MANOUSH ZOMORODI, HOST:

On the show today, reshaping evolution – how recent medical advances may allow us to defeat some of today’s most debilitating illnesses.

NABIHA SAKLAYEN: Imagine you’re having some strange symptoms, and you don’t really know what they are.

ZOMORODI: This is Nabiha Saklayen.

SAKLAYEN: You’re feeling a lot of tremors. Your muscles are stiff. You’re having difficulty thinking and understanding. And you go to your doctor because you want to know what’s going on.

ZOMORODI: And they run tests – a whole host of them. They order neurological exams, look at your family history, measure your agility, muscle tone, balance. And then…

SAKLAYEN: And then you find out that you have Parkinson’s disease, which is devastating news. And I’ve had family members who’ve had to go through that. And it’s a very, very tough moment to realize you have something that’s going to completely change your life, and you might have these symptoms forever.

ZOMORODI: Today, your doctor would review your options with you – medications you can take, lifestyle changes to make, how to manage a disease which has no cure.

SAKLAYEN: But…

ZOMORODI: But Nabiha is working towards a different outcome.

SAKLAYEN: What if I told you there is a different future ahead for us? What if your doctor, instead of saying, OK, we’re going to be treating your symptoms, your doctor actually says, no, we’re going to be able to cure this disease? And the way that works is you drop off a blood sample. Those blood cells are shipped off to a cell factory to generate brand new neurons that are customized just for you. You come back the next week, and a surgeon transplants those neurons into your brain, and you just received a cure for Parkinson’s.

ZOMORODI: OK, wait, let me see if I get this right. Take some blood cells, turn them into new neurons, put them in the right places in the damaged part of the brain, and you can essentially cure Parkinson’s.

SAKLAYEN: Exactly. Yeah.

ZOMORODI: I mean, Nabiha, I got to say, it sounds like science fiction.

SAKLAYEN: It does sound like science fiction, but what’s amazing is, most of the pieces of the puzzle have been figured out. We know how to make patient-specific cells. We know how to transplant them into the right part of the brain. We’ve seen these transplants happen in patients. And the results are very, very promising. And now we just have to figure out manufacturing. How do we make these cells in a fully automated way, make them super cheap? And that’s what I’m working on with my team.

ZOMORODI: For years, we’ve heard that stem cells may eventually cure diseases and treat illnesses – that by genetically engineering them, they could fix our ailing bodies. Now, though it’s still early, promising new technologies are getting us closer, in labs like those at Nabiha’s company, Cellino.

SAKLAYEN: We’re automating the generation of personalized human cells. And these cells can be used for a range of therapeutic applications.

(SOUNDBITE OF TED TALK)

SAKLAYEN: And because they’re 100% your cells, your immune system is extremely unlikely to reject or attack those cells.

ZOMORODI: Nabiha Saklayen picks up this idea from the TED stage.

(SOUNDBITE OF TED TALK)

SAKLAYEN: In fact, the body has no idea that these cells were actually made in a cell factory. All of this is possible because of a breakthrough at the intersection of biology, laser physics and machine learning. We’ll start with biology. The human body is an absolute miracle. Trillions of cells are working in synchronicity to pump blood, secrete dopamine and let me see and speak to you right now. But as we age, our cells age, too. That’s why our skin starts to sag, our cartilage wears away and your five-mile run might turn into a 20-minute walk. Yes, we’re all getting older. Our bodies are ticking time bombs. But stem cells could offer a solution.

ZOMORODI: All right. So stem cells – remind us, why are they so useful?

SAKLAYEN: Yes. Stem cells are these very special cell types that have the code in them to become any cell type in the body. In our natural state of existence, we don’t have absolutely 100% pure stem cells in the body anymore because they’ve evolved into becoming different subsets of cells in the body. But it’s possible now to generate really high-quality stem cells for each and every patient, for each and every adult, that look very much like embryonic cells.

ZOMORODI: Scientists can make stem cells.

SAKLAYEN: Absolutely. They’re called induced pluripotent stem cells, and these stem cells open up the possibility to generate neurons on demand, heart cells on demand, skin on demand, hair on demand. You name it. You can make any cell type…

ZOMORODI: Wow.

SAKLAYEN: …Where we know the biology of how to change the stem cell to the target cell type.

(SOUNDBITE OF TED TALK)

SAKLAYEN: Now, unfortunately, stem cells are notoriously difficult to engineer. One fundamental problem relates to how they’re made, which involves taking a patient’s blood cells and adding chemicals to those blood cells to turn them into stem cells. Now, during this chemical process, you never end up with a perfect set of stem cells. In fact, you get a very messy plate of cells going in different directions – towards the eye, brain, liver. And every random cell must be removed. Until recently, the main way to remove cells was by hand. I remember the first time I visited the Harvard Stem Cell Institute. I watched a highly skilled scientist sitting at a bench, looking at stem cells, evaluating them one at a time and removing the unwanted cells by hand. It’s a slow, tedious and artisanal process, which is why generating a personalized stem cell bank today costs about $1 million.

Right now, there are phase one/two clinical trials – one that’s already launched in the U.S. – for personalized iPSC-based therapies. All of these groups have made enough patient-specific stem cells and therapies and derived tissues ***

SAKLAYEN: *** by hand. And that’s maybe 10, 15 or 20 patients.

ZOMORODI: That’s it.

SAKLAYEN: That’s it. Right. So when you think about a phase three trial, you may need hundreds of patients, and there literally aren’t enough scientists that can make those cells by hand. And, of course, it becomes very, very expensive. Running a phase three clinical trial would cost $300 million, which is not…

ZOMORODI: Wow.

SAKLAYEN: …Feasible in most instances.

ZOMORODI: OK. And this is where, presumably, your work comes in, Nabiha.

SAKLAYEN: Yes. So when I came into the biology space – so just a quick background. I’m a physicist by training.

(SOUNDBITE OF TED TALK)

SAKLAYEN: When I started my Ph.D., I joined a laser physics lab because lasers are the coolest. But I also decided to dabble in biology. I started using lasers to engineer human cells. And when I talked to biologists about it, they were amazed. Here’s why – scientists are always looking for ways to make biology more precise. Sometimes cell culture can feel a lot like cooking. Take some chemicals, put it in a pot, stir it, heat it, see what happens, try it all over again. In contrast, lasers are so precise. You can target one cell in millions at precise intervals every second, every minute, every hour. I realized that instead of doing this tedious process of stem cell culture by hand, we could use lasers to remove the unwanted cells. And to automate the entire process, we decided to use machine learning to identify those unwanted cells and zap them.

Here’s how it works – take some blood cells, put it in a cassette, add chemicals to those blood cells to turn them into stem cells, like always. Now, instead of having a human look for those unwanted cells and remove them by hand, the machine identifies the unwanted cells and zaps them with a laser. As you can see, this entire process happens by machine. The computer decides when and how often to prune the cells and uses a fully automated system to run the process. After repeated pruning, you end up with a perfect culture of your stem cells ready to be banked and used at any time.

ZOMORODI: Nabiha, we talked about how, hypothetically, a brain with Parkinson’s could be repaired using stem cells – IPSCs. But to be clear, stem cells are already being used to treat leukemia and other types of blood cancers. I’ve also read about them being used to restore a patient’s eyesight in clinical trials. So so far, it seems pretty promising.

SAKLAYEN: Yes. And just this past month, there’s been an amazing result that was put out into the world by the Vertex team where they tested one patient with a dose of new insulin-producing cells. And this patient is not having to use insulin injections anymore. So that has happened in the past month, and it’s tremendous. And now it’s all about figuring out what is the right format for the specific cell therapy, how many cells should be transplanted, how to get around the immune evasion problems and how to manufacture these cells in a scalable and cost-effective way. That’s what we need to figure out. But we’re going to do all of that in the next 10 years. I have so much confidence we are as an industry.

(SOUNDBITE OF TED TALK)

SAKLAYEN: Perhaps you have longevity in mind. That is certainly a possibility. In the future, we might use these exact same stem cell banks to generate entire new organs, new tissues, new skin, new bone. This technology also has the potential to revolutionize personalized pharmaceuticals. Today, taking medicine is, to some degree, trial and error. You don’t really know if the drug is going to work for you until you put it in your body. But what if we had a miniature human replica of you with your cells? Eye cells, brain cells, heart cells, muscle cells, blood cells on a chip – a miniature human replica of you. We could take the drugs, test them on the cells in the lab first to see how it works. If it works, fantastic, go ahead and take the drug; if it doesn’t, pharmacists can order up custom drugs just for you.

ZOMORODI: You know, there was a time that if you were diagnosed with smallpox, I mean, that was it. It was fatal. But now, of course, smallpox is gone. It’s been eradicated. In the future, do you think that – is that how we’re going to think about diseases like Parkinson’s and diabetes? If or when we have these stem cell treatments, will these illnesses be so easy to cure that a diagnosis won’t really feel as devastating or life altering as it does now?

SAKLAYEN: I do think there is a possibility to create a world where these diseases don’t feel as burdensome as they are today. However, I do want to mention, also, this brings me back to a lot of thoughts I have about accessibility in health care and how do we make these cell therapies accessible? It really comes down to how cheap can we make these advanced therapies? You know, getting a cell therapy could be just as expensive as buying insulin or taking penicillin or taking painkillers. That would be what is my aspirational goal for the future. But we have so much, so much work to do to get there because making cells is so complex. But I am very optimistic we’re going to get there because what’s happening right now in bioengineering is many different disciplines are coming together and trying to solve these big problems in new and creative ways.

ZOMORODI: Nabiha Saklayen is the co-founder and CEO of Cellino Biotech. On the show today, reshaping evolution. I’m Manoush Zomorodi, and you’re listening to the TED Radio Hour from NPR. Stay with us.

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